NICE won't back Novartis' myelofibrosis drug

NICE is not backing Novartis’s Jakavi after its found problems with the firm’s data and costing model for its drug.

The pricing watchdog has said in new draft guidance that it is not recommending Novartis’ Jakavi (ruxolitinib) for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adults with primary myelofibrosis, or myelofibrosis secondary to polycythaemia vera or essential thrombocythaemiai.

NICE’s Committee said that although it was plausible Novartis’ drug could offer a survival benefit, it noted “uncertainties in the data” used by the manufacturer to estimate the degree of survival benefit.

The Committee also said that there were “fundamental issues” with Novartis’ costing model, making its cost-effectiveness uncertain.

Novartis believes that its drug is worth £74,000 per QALY gained, but NICE argues that it is actually double this – NICE does not usually recommend a drug that has a QALY over £30,000.

Jakavi costs £3,600 for a 60-tablet pack of 15 mg or 20 mg tablets, or £1,800 for a 60-tablet pack of 5 mg, meaning an annual cost of around £43,200 per patient. The firm has not offered a patient access scheme to help lower the cost of the medicine.

NICE concluded that Jakavi was clinically effective and a step change in innovation for treating the condition, but could not be considered a cost-effective use of NHS resources compared with best available therapy for treating disease-related splenomegaly or symptoms in adults with myelofibrosis

Professor Carole Longson, health technology evaluation centre director at NICE, said: “Myelofibrosis and splenomegaly can be extremely debilitating, with symptoms such as severe itching and fatigue.

“It is disappointing not to be able to recommend this new treatment in our preliminary recommendations, but in order to do this we have to be sure that the treatment is both clinically and cost effective, because money has to be diverted from elsewhere in the health service to pay for it. The draft guidance is now out for consultation and I would urge all those with an interest in myelofibrosis to comment via the NICE website.”

Myelofibrosis is a type of haematological (blood) cancer and is a rare condition, with around 0.4 cases per 100,000 each year in the UK. The bone marrow becomes scarred (fibrosed) making it is less able to make blood cells.

To compensate for this, other organs in the body, including the liver and the spleen, begin to produce them. As the spleen begins to produce blood cells, it grows in size causing splenomegaly.